Biggest FDA Developments for 2017

As we begin the new year, it is time toassess the previous year and consider what really mattered when all is said and done. We’ve perhaps already done this on a personal level, but here is my take on an FDA level.

The fact that more is happening has been reflected perhaps by the issuance of many more agency press releases this year by far over last year – 164 for 2017 compared to 122 for 2016. Many of the releases this year were special statements by the Commissioner to provide additional commentary on developments at the agency.

And so among all that activity, here comes my pick for the top five developments at/by FDA for this past year.

1. Approvals in Cancer Treatments – Reviewing the list of approvals of new molecular entities for 2015, FDA has approved 12 new cancer-related treatments which is 8 more than were announced by FDA in 2016 and nearly as many as were approved in the banner year of 2015. It is not just the number of cancer treatment approvals, but the scope as well. There were a large number of approvals aimed at types of blood based cancers – AML/leukemia as well as breast cancer and the approval of the first biosimilar for use in breast and stomach cancer. In addition, the agency granted accelerated approval for a treatment indicated for any solid tumor that has a specific genetic feature – the first time an approval was based not on the location of the tumor, but rather on a common biomarker.

2. CAR-T Approval – Related to the above, not a drug treatment but a cell-based gene therapy, the approval of the first CAR-T Therapy in August of this year merits its own category given the degree to which it evolves cancer treatment. A long-sought goal of cancer therapy has been to unleash the ability of the immune system itself to resist the cancer. CAR-T manages just that. While the earliest work has been successful in blood borne cancers, there is reason to hope that there might one day be used in solid tumors. Each dose is customized and is created using the patient’s own T-Cells, genetically modified to include a new gene that targets and kills cancer cells and is then re–introduced to the patient. The August approval was for the treatment of acute lymphoblastic leukemia (ALL) and the second approval in October was for certain types of B-Cell lymphoma. CAR-T represents a big change.

3. Digital Health Package – While FDA has been extremely slow in anticipating and adapting to developments in social and digital media from a communications point of view, the agency has been much more responsive to developments in digital health. In July the agency announced a framework to facilitate the development and approval of digital health devices by releasing the Digital Health Innovation Action Plan which included a program for developers of devices and software to be pre-certified, shifting from a focus solely on the device to the entity that is developing it, characterizing the balance that FDA is seeking to strike between fostering innovation in fast-paced sector with ensuring high quality and safety of products. The companies chosen for pre-certification were announced in September, and FDA will be holding a two-day public meeting later this month .

4. Regenerative Medicine Policy – “We’re at the beginning of a paradigm change in medicine with the promise of being able to facilitate regeneration of parts of the human body, where cells and tissues can be engineered to grow health, functional organs to replace diseased ones; new genes can be introduced into the body to combat disease; and adult stem cells can generate replacements for cells that are lost to injury or disease. This is no longer the stuff of science fiction. This is the practical promise of modern applications of regenerative medicine.” These were the words of Commissioner Gottlieb as FDA announced its comprehensive regenerative medicine policy with the issuance of four final and draft guidance documents designed to add clarity to FDA’s regulation of this space. The objective of the policy is to move FDA forward, as in other areas, in taking a risk-based approach to regulation. At the same time, earlier in the year FDA issued a warning to a stem cell practitioner and has a web page dedicated to helping consumers understand the potential harms that may exist with unapproved approaches.

5. Confirmation of Dr. Scott Gottlieb as Commissioner – Since assuming his post, the new Commissioner of the Food and Drug Administration, Dr. Gottlieb has demonstrated both breadth and depth when it comes to taking charge of this mammoth agency. Over the course of his initial months he has set into motion a range of activity on multiple fronts that includes various aspects of dealing with the opioid epidemic, seeing to easing the path for generic drugs, two issues that were of particular concern during his confirmation process. As noted above, he has also begun processes that are designed to facilitate the development of new types of devices and to address the changing landscape of treatment.

Beyond the scope of activity is the impact. There is a real attempt at implementing a cultural change and mindset of the agency – from doing things the way that things have traditionally been done to comprehensively laying out a path to a risk-based approach to regulatory oversight, focusing resources on those areas where the greatest risk is involved with the objective of enhancing innovation while maintaining a balance with safety and efficacy, and not just in drugs and devices, but across the board. While the 21st Century Cures Act has been a legislative mandate along those lines, he has long been a proponent of a risk-based approach and his tenure at FDA is very likely to leave a large footprint. Finally, not only has he set out to put policy into motion, he is taking expanded steps to communicate the change not only through frequent blog posts, but as recently noted in a posting here on Eye on FDA, by issuing clarifying statements in addition to agency press releases that detail for stakeholders the steps that the agency is taking.

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About the Author

My name is Mark Senak. I’m a lawyer and I work at the international communications firm FleishmanHillard in New York. For the past several years, I have been consulting with pharmaceutical and biologic companies that are engaged in the process of bringing new drugs to market.

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I am an authority on regulatory aspects of communications and medical products, with particular emphasis on pre-approval communications; strategist to help pharma and biotech companies prepare best case for advisory committee approval; and counselor in issues and crisis management. I am a frequent speaker on various aspects of same - drug development, promotion, reimbursement and new media in a highly regulated environment. Author of books, newspaper and magazine pieces related to drug marketing and promotion as well as HIV specialty pieces. And of course... blogger!

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Eye on FDA is published by Mark Senak of FleishmanHillard's Washington, D.C. office. The thoughts and ideas in this blog and postings are strictly my own and are not screened by my employer. Everything posted on this blog is my personal opinion and does not necessarily represent the views of FleishmanHillard or its clients.